First Treatment for all patients with Duchenne approved

This week we received THE BEST NEWS the first treatment for EVERYONE living with Duchenne has been approved!!!!  NICE, the National Institute of Clinical Excellence in England and Wales announced that Agamree (also known as Vamorolone) a dissociative steroid, has been approved for ALL patients with Duchenne Muscular Dystrophy. Patients need to be above the age of 4. But critically, in a UK first, the treatment has been made available to. ALL patients regardless of mutation and to all patients – those who can walk and those who cannot. There is no upper age limit for treatment. And that’s the first time this has happened.
Vamorolone, a new steroids-alternative drug had been a focal point for the Duchenne community for many years!!!
Duchenne UK has been a big player in making this happen. Many years ago Alex’s Wish gave £47,000 towards the very early stages working alongside Dr Eric Hoffman.
We at Alex’s Wish feel incredibly proud to have played a part in making this possible 💙💙💙
A huge thank you to all of our incredible supporters for your continued help and support. It is so great to demonstrate another milestone has being achieved. Thank you 👏🙏🙌

Posted in Drug approval process